The Neuromuscular Program at Stanford and Lucile Packard Children’s Hospital (LPCH) will hold the 3rd Annual Spinal Muscular Atrophy Educational Conference at California’s Stanford School of Medicine on Saturday, May 13.
The conference, designed for spinal muscular atrophy (SMA) patients and caregivers, will take place from 10 a.m. to 3:30 p.m. at Stanford’s Li Ka Shing Center, at 291 Campus Drive, Stanford, CA 94305. It will focus on the newly approved drug Spinraza (nusinersen) and include seminars on maintaining benefits while working, independent living strategies, physical therapy, exercise and more.
Spinraza, which is injected into the spinal fluid once every few months to reach the nerves where it is needed, received U.S. Food and Drug Administration (FDA) approval in December 2016. The Stanford SMA program has helped investigate Spinraza’s effects as well as possible new treatments. Stanford and LPCH, which have been studying the drug for more than four years, now offer weekly multi-disciplinary clinics for infants, children and adults with SMA.
Zoe was the first patient to receive Spinraza at Stanford. Before she began receiving the drug, seven-month-old Zoe — who has SMA type 1 — couldn’t sit up or roll over. She couldn’t move her legs at all, or lift her arms when she was lying down. She struggled to swallow. Zoe then entered a Phase 2 clinical trial to receive the then-experimental drug Spinraza. The next year was difficult. Whenever she seemed to grow stronger, another bout of illness weakened her again.
Finally, near Zoe’s second birthday, everyone was convinced that she was gaining strength. Remarkably for a toddler with SMA-1, she could lie on her back, lift her legs and play with her toes.
The conference will include a question-and-answer discussion with experts; please click here to register.
Attendees do not have to be a part of the Stanford or LPCH community. Lunch is included, but participants must pre-register by April 28. Childcare isn’t offered at this event due to staffing constraints. For more information, please contact Jennifer Fisher at JNFisher@Stanford.edu or (650) 497-5934.